[HTML][HTML] A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

S Hacein-Bey-Abina, C Von Kalle… - New England journal …, 2003 - Mass Medical Soc
S Hacein-Bey-Abina, C Von Kalle, M Schmidt, F Le Deist, N Wulffraat, E McIntyre, I Radford…
New England journal of medicine, 2003Mass Medical Soc
To the Editor: We recently reported (April 18 issue) 1 the sustained correction of X-linked
severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the
γ c gene into CD34+ cells in four of five patients with the disease. These results have since
been confirmed in four additional patients with typical X-linked severe combined
immunodeficiency. Of the first four successfully treated patients, three continue to do well up
to 3.6 years after gene therapy, whereas a serious adverse event occurred in the fourth …
To the Editor: We recently reported (April 18 issue)1 the sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease. These results have since been confirmed in four additional patients with typical X-linked severe combined immunodeficiency. Of the first four successfully treated patients, three continue to do well up to 3.6 years after gene therapy, whereas a serious adverse event occurred in the fourth patient. At a routine checkup 30 months after gene therapy, lymphocytosis consisting of a monoclonal population . . .
The New England Journal Of Medicine